hATTR-PN

Adults with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), lived longer after treatment with tafamidis versus undergoing a liver transplant, a study reported. However, patients treated with tafamidis experienced a faster decline in neurological and heart-related outcomes than did those who…

Most people with late-onset familial amyloid polyneuropathy (FAP) experience pain as a result of nerve damage, referred to as neuropathic pain, a recent study highlights. Findings suggest that neuropathic pain is linked to worse life quality, and many patients do not experience relief from this type of pain even…

Sudoscan, a commercial device that evaluates sweat gland function, might be a useful noninvasive tool to assess damage to small nerve fibers in people with familial amyloid polyneuropathy (FAP), a small study showed. Those small nerve fibers usually are affected in the early stages of the disease, leading to symptoms like…

Caring for a person with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has a considerable impact on caregivers’ health-related quality of life (HRQoL) and productivity, a survey reported. The findings were in the study, “A survey exploring caregiver burden…

Tegsedi (inotersen) helped to stabilize familial amyloid polyneuropathy (FAP) symptoms in a 71-year-old man, but its use also was associated with a rare case of liver damage after one year of treatment. His liver enzymes, a marker of damage to the organ, rose well beyond the normal range, scientists reported.

England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available through the country’s National Health Service (NHS) for treating familial amyloid polyneuropathy (FAP). Amvuttra is one of the first rare disease treatments to get the green light from NICE…

Eye involvement can become common over time in people with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), a study from Spain reported. Researchers also noted that symptoms affecting the eyes are often slower to develop than others due to this disease. As…

I have previously written about the importance of staying positive despite our circumstances. Whether you are a patient or a caregiver to a loved one with a chronic illness, keeping yourself above the waterline to avoid drowning in your circumstances is essential. As the legendary basketball player Michael…

An elderly man in Germany was found to carry one copy of a new mutation in the TTR gene that caused both neurological and heart symptoms of familial amyloid polyneuropathy (FAP), which were described in a recent case report. The man’s case calls for “careful molecular genetic analysis and…

Quantitative sensory testing (QST), a tool for monitoring nerve function, may help to detect problems in small nerve fibers in people with late-onset familial amyloid polyneuropathy (FAP) before symptoms start, a small study suggested. Adults with disease-causing mutations for late-onset FAP but no disease symptoms had more abnormal responses…